Sight Restored: Gene Therapy Breakthrough Wins Major Science Prize
A married couple and investigator Katherine High share a $3 million Breakthrough Prize for Luxturna, the gene therapy approved in 2017 that restores vision in people with Leber congenital amaurosis by delivering a working RPE65 gene to retinal cells; the 25-year project produced dramatic patient improvements and was celebrated at a Los Angeles ceremony, while scientists warned about politicized attacks on science and other prize-winning work highlighted advances in gene therapies for blood disorders.